Here you can read a selection of notable comments about the landmark paper on in vivo genome-editing published in the New England Journal of Medicineon 26 June. The trial, conducted in the UK and New Zealand, produced the first-ever clinical data supporting the safety and efficacy of intravenous infusion of a single-dose CRISPR treatment. The treatment, developed by two US-based companies (Intellia Therapeutics and Regeneron Pharmaceuticals) targets a rare and fatal condition called transthyretin amyloidosis.
Jennifer Doudna (CRISPR co-inventor and co-founder of Intellia): “It’s a critical first step in being able to inactivate, repair, or replace any gene that causes disease, anywhere in the body” (source Science).
According to his Twitter bio, Charles Murray is a “Husband, father, social scientist, writer, Madisonian. Or maybe right-wing ideologue, pseudoscientist, evil. Opinions differ.” You may remember him as the co-author of the controversial book “The Bell Curve” (1994), discussing purported connections between race and intelligence. The bad news is that he recently joined the CRISPR debate by tweeting “Gene editing to raise IQ will have a huge market”. The good news is that confutation is easy and a little irony is the best reply (check out Fyodor Urnov’s tweet in the gallery below).
Hopefully, CRISPR-based diagnostics will make an early debut amid COVID-19 outbreak. But what about a CRISPR prophylactic strategy to combat coronaviruses? The proof of concept is here, in bioRxiv, but it will be deployed in the next pandemic if we are lucky. It’s called PAC-MAN, like the videogame, stands for Prophylactic Antiviral CRISPR in huMAN cells, and comes from the Stanley Qi Lab.
The year 2019 ended with three years in jail sentenced to He Jiankui for illegal medical practice. The CRISPR-baby scandal’s epilogue was applauded on twitter by a few leading scientists such as Craig Venter and Fyodor Urnov and decried on STAT News by the controversial biohacker Josiah Zayner. Most experts, however, stayed silent.
As stressed by the Washington Post, “the judicial proceedings were not public, and outside experts said it is hard to know what to make of the punishment without the release of the full investigative report or extensive knowledge of Chinese law and the conditions under which He will be incarcerated.”
Doudna meets GSK: University of California CRISPR researchers form drug discovery alliance with pharma giant (source Science) From transposons to gene therapy: Hijack of CRISPR defences by selfish genes holds clinical promise, according to Fyodor Urnov (NatureNews&Views) Disaster waiting to happen: Russian biologist plans more CRISPR-edited babies (Nature’s editorial and news)